Bezeparsen

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Tue Apr 01 22:56:22 GMT 2025` Edited by `admin` on `Tue Apr 01 22:56:22 GMT 2025`
Nucleic Acid Type	OLIGONUCLEOTIDE
Sequence Type	COMPLETE
Record UNII	CP66E80XXT
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

DNA, d(P-thio)([2?,5?-anhydro-6?-deoxy-4?-C-(hydroxymethyl)-?-L-mannofurano]rA-(3??4?)-[2?,5?-anhydro-6?-deoxy-4?-C-(hydroxymethyl)-?-L-mannofurano]rA-(3??4?)-[2?,5?-anhydro-6?-deoxy-4?-C-(hydroxymethyl)-?-L-mannofurano]m5rU-A-A-T-m5C-T-m5C-A-T-G-T-(3??4

Preferred Name

English

Bezeparsen

Official Name

English

all-P-ambo-5'-O-(28-[(2-acetamido-2-deoxy-?-D -galactopyranosyl)oxy]-16,16-bis{[3-({6-[(2-acetamido-2-deoxy-?-D -galactopyranosyl)oxy]hexyl}amino)-3-oxopropoxy]methyl}-1-hydroxy-1,10,14,21-tetraoxo-2,18-dioxa-9,15,22-triaza-1? 5 -phosphaoctacosan-1-yl)-2

Common Name

English

bezeparsen [INN]

Common Name

English

Code System Code Type Description

NCI_THESAURUS

C190429

Created by admin on Tue Apr 01 22:56:22 GMT 2025 , Edited by admin on Tue Apr 01 22:56:22 GMT 2025

PRIMARY

FDA UNII

CP66E80XXT

Created by admin on Tue Apr 01 22:56:22 GMT 2025 , Edited by admin on Tue Apr 01 22:56:22 GMT 2025

PRIMARY

CAS

2639640-84-7

Created by admin on Tue Apr 01 22:56:22 GMT 2025 , Edited by admin on Tue Apr 01 22:56:22 GMT 2025

PRIMARY

INN

12322

Created by admin on Tue Apr 01 22:56:22 GMT 2025 , Edited by admin on Tue Apr 01 22:56:22 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
	1_1-1_3;1_14-1_16	6 / 16
Deoxyribose (DNA)	1_4-1_13	10 / 16

Linkage	Site Range	Site Count
Phosporothioate	1_1-1_15	15 / 15

Related Record Type Details


					LGW95S5DG1

PROPROTEIN CONVERTASE SUBTILISIN/KEXIN TYPE 9

TARGET->INHIBITOR OF EXPRESSION

Amount:

Related Record Type Details


					CP66E80XXT

Bezeparsen

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Fragment Name	Fragment Approval
NUCLEOSIDE_SUBSTITUTION	[1_1]	5'-TERMINUS	UNSPECIFIED SUBSTANCE	551bcbdf (not in database)
NUCLEOSIDE_SUBSTITUTION	[1_3]	SITE_SPECIFIC	2'-O,4'-C-((1S)-ETHANE-1,1-DIYL).BETA.-D-RIBOSE 5-METHYL URIDINE	I12206O08P
NUCLEOSIDE_SUBSTITUTION	[1_7] [1_9]	SITE_SPECIFIC	5-METHYLDEOXYCYTIDINE	B200GV71QM
NUCLEOSIDE_SUBSTITUTION	[1_14]	SITE_SPECIFIC	2'-O,4'-C-((1S)-ETHANE-1,1-DIYL).BETA.-D-RIBOSE 5-METHYL CYTIDINE	7XRE49320R